Repurposed DFMO Could Bring Hope for Bachmann-Bupp Syndrome
Decades-old DFMO (eflornithine), an inhibitor of the ODC1 enzyme, is being explored as a therapy for Bachmann-Bupp syndrome, an ultra-rare neurodevelopmental disorder. Early use in a handful of patients—some via FDA-approved single-patient protocols—has shown symptom improvements. A collaboration among Corewell Health, Michigan State University, and Every Cure aims to build preclinical data and streamline regulatory paths toward a formal clinical trial, addressing awareness, study design, and endpoints. With about 20 cases identified worldwide, the group plans a preclinical study next year to bolster evidence and help bring potential DFMO therapy to more patients.